ProQR Therapeutics is a biotechnology company active in development and delivery of transformative RNA medicines for the treatment of severe diseases such as cystic fibrosis and Leber’s congenital amaurosis. The company is developing a therapy for cystic fibrosis with the aim the release patients from their symptoms by resolving the underlying genetic defect. Cystic fibrosis is a genetic orphan disease affecting 90.000 patients worldwide.

The company is led by a team of technology, drug development and disease experts and supported by industry icons.

Since Gilde invested in ProQR in 2014, the company has advanced QR-010 through preclinical development for patients with cystic fibrosis. ProQR expanded its platform and pipeline with QR-110 for the treatment of Leber’s Congenital Amaurosis (LCA), the leading genetic cause of blindness in childhood. ProQR successfully completed an IPO on the NASDAQ Global Market in September 2014 resulting in aggregate net proceeds, of approximately $102 million.

ProQR Therapeutics is located in Leiden (the Netherlands).